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The 2023-2028 Outlook for Rare Disease Drugs in the United States

October 2022 | 503 pages | ID: 2FB55F40DF58EN
ICON Group International

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This study covers the latent demand outlook for rare disease drugs across the states and cities of the United States. Latent demand (in millions of U.S. dollars), or potential industry earnings (P.I.E.) estimates are given across some 12,600 cities in the United States. For each city in question, the percent share the city is of its state and of the United States as a whole is reported. These comparative benchmarks allow the reader to quickly gauge a city vis-à-vis others. This statistical approach can prove very useful to distribution and/or sales force strategies. Using econometric models which project fundamental economic dynamics within each state and city, latent demand estimates are created for rare disease drugs. This report does not discuss the specific players in the market serving the latent demand, nor specific details at the product level. The study also does not consider short-term cyclicalities that might affect realized sales. The study, therefore, is strategic in nature, taking an aggregate and long-run view, irrespective of the players or products involved.

In this report we define the sales of rare disease drugs as including all commonly understood products and/or services falling within this broad category, irrespective of product packaging, formulation, size, or form. Companies participating in this industry include AB Science, Acceleron Pharma & Celgene, Acer Therapeutics, Actavis Generics, Actelion Pharmaceuticals, Actinium Pharmaceuticals, Adaptimmune, Aduro BioTech, Advanced Accelerator Applications, Advantagene, Advaxis, Advenchen Laboratories, Affimed Therapeutics & Merck, Agenus, Agios Pharmaceuticals, Akashi Therapeutics, Akcea Therapeutics, Alcobra, Alexion Pharmaceuticals, Alkeus Pharmaceuticals, Allergan, Alnylam Pharmaceuticals, Ambit Biosciences, AmerisourceBergen Corporation, Amgen, Amicus Therapeutics, Andarix Pharmaceuticals, Angimmune, AngioChem, AntiVirus Therapeutics, Applied Genetic Technologies, Araim Pharmaceuticals, Arch Biopartners, arGentis Pharmaceuticals, Ariad Pharmaceuticals, Armagen Technologies, ARMGO Pharma, AROG Pharmaceuticals, ArQule, Array BioPharma, Arrowhead Research, Astellas Pharma, Astex Pharmaceuticals, AstraZeneca, Atara Biotherapeutics, Atox Bio, aTyr Pharma, Baxalta, Bayer Healthcare, Belrose Pharmaceuticals, BerGenBio, Bioblast Pharma, BioCryst Pharmaceuticals, Biodel, Biogen, BioInvent International, BioLineRX, BioMarin Pharmaceutical, BioNTech, Bio-Path Holdings, Biotest Pharmaceuticals, Blaze Bioscience, Bluebird Bio, Blueprint Medicines, Boehringer Ingelheim, Bristol-Myers Squibb, Burzynski Research Institute, Caladrius Biosciences, Cancer Advances, Capricor Therapeutics, Cardinal Health, CASI Pharmaceuticals, Catabasis Pharmaceuticals, Cavion, Celator Pharmaceuticals, Celgene Corporation, Cell Medica, CEL-SCI, CELSION, Celtaxsys, Cempra Pharmaceuticals, CerRx, Cerulean Pharma, ChemoCentryx, Chugai Pharma, Cidara Therapeutics, Cleave Biosciences, Clementia Pharmaceuticals, Clinuvel, Clovis Oncology, Confluence Pharmaceuticals, Corbus Pharmaceuticals, Cornerstone Pharmaceuticals, Cortice Biosciences, Critical Outcome Technologies, Curis, Cyclacel, CymaBay Therapeutics, CytRx, Daiichi Sankyo, Deciphera Pharmaceuticals, Delcath Systems, DelMar Pharmaceuticals, Diffusion Pharmaceuticals, Dimension Therapeutics, Diurnal, DNAtrix, Eddingpharm, Edimer Pharmaceuticals, Edison Pharmaceuticals, Eiger BioPharmaceuticals, Eisai, Eleison Pharmaceuticals, Eli Lilly, EMD Serono & Pfizer, Emergent BioSolutions, Emmaus Medical, Epitopoietic Research, Epizyme, EryDel, Escala Therapeutics, Essentialis, Five Prime Therapeutics, Fortress Biotech, Gamida Cell, Gem Pharmaceuticals, Gen Sight Biologics, GenSpera, Genzyme, Gilead Sciences, GlaxoSmithKline, Gliknik, Global Blood Therapeutics, GlycoMimetics, Golden Biotechnology, Gradalis, Grifols Therapeutics, GW Pharma, Halozyme Therapeutics, Hanmi, Hoffmann-La Roche, Horizon Pharma, Idera Pharmaceuticals, Ignyta, Immatics Biotechnologies, Immune Design, Immunocellular Therapeutics, Immunogen, Immunomedics, Immunovaccine Technologies, Incyte, Infinity Pharmaceuticals, InnoPharma, Insmed, Insys Therapeutics, Ionis Pharmaceuticals, Ipsen Biopharmaceuticals, IRX Therapeutics, Janssen Biotech, jCyte, Juno Therapeutics, Karyopharm Therapeutics, Kevelt, Kyowa Kirin USA, LeafBio, Lexicon Pharmaceuticals, Lion Biotechnologies, LipimetiX Development, MabVax Therapeutics, Marathon Pharmaceuticals, Marina Biotech, Mast Therapeutics, McKesson Corporation, MedImmune, MEI Pharma, Merck & Company, Millendo Therapeutics, Millennium Pharmaceuticals, Milo Biotechnology, Minneamrita Therapeutics, Mirati Therapeutics, Momenta Pharmaceuticals, MorphoSys, Nektar Therapeutics, NeOnc Technologies, Neuren Pharmaceuticals, Neurolixis, Neurotech USA, NGM Biopharmaceuticals, NightstaRx, Nivalis Therapeutics, NKT Therapeutics, Novartis Pharmaceuticals, Novo Nordisk, Ocata Therapeutics, Omeros, On Target Laboratories, Oncolytics Biotech, OncoMed Pharmaceuticals, Onconova Therapeutics, OncoPep, Opko Biologics, OSE Pharma, Otsuka Pharmaceutical, OXiGENE, Patagonia Pharmaceuticals, Peregrine Pharmaceuticals, Pfizer, Pharmacyclics, PharmaCyte Biotech, PharmaEssentia, Corporation, PharmaMar USA, Plexxikon, PNP Therapeutics, Polaris Pharmaceuticals, Polynoma, Prana Biotechnology, Precision Biologics, Progenics Pharmaceuticals, Prolong Pharmaceuticals, Promedior, ProMetic Biotherapeutics, ProNAi Therapeutics, ProQR Therapeutics, Protalex, Provectus Pharmaceuticals, PTC Therapeutics, QLT, Quest PharmaTech, Qurient, Raptor Pharmaceutical, Rebiotx, Regeneron Pharmaceuticals, Regulus Therapeutics & Sanofi US, Retrophin, RetroSense Therapeutics, ReveraGen Biopharma, Rexahn Pharmaceuticals, Rhythm Metabolics, Rigel Pharmaceuticals, River Vision, Roche, Sanofi, Santhera Pharmaceuticals, Sarepta Therapeutics, Savara Pharmaceuticals, Seattle Genetics, Selexys Pharmaceuticals, SELLAS Life Sciences, Sequella, Seres Health, Shire, SIGA Technologies, Sigma-Tau Pharmaceuticals, SillaJen Biotherapeutics, Soligenix, Soricimed Biopharma, Spark Therapeutics, StemcentRx, Stemline Therapeutics, Sun Biopharma, Takeda Pharmaceuticals, Tapimmune, Tarix Orphan, Tesaro, TetraLogic Pharmaceuticals, Teva Pharmaceutical Industries, TG Therapeutics, Tiziana Life Sciences, Tocagen, Tolero Pharmaceuticals, Tracon Pharmaceuticals, TransDerm, Triphase Research & Development, Ultragenyx Pharmaceutical, UniQure, UroGen, Vascular Biogenics, VentiRx Pharmaceuticals, Verastem, Vertex Pharmaceuticals, Viamet Pharmaceuticals, Vicus Therapeutics, Viralytics, Viventia Biotech, VivoLux, Vtesse, Wilson Therapeutics, Xeris Pharmaceuticals, XOMA, Ziopharm Oncology, Zogenix, and Zywie. In addition to the sources indicated, additional information available to the public via news and/or press releases published by players in the industry was considered in defining and calibrating this category. All figures are in a common currency (U.S. dollars, millions) and are not adjusted for inflation (i.e., they are current values). Exchange rates used to convert to U.S. dollars are averages for the year in question. Future exchange rates are assumed to be constant in the future at the current level (the average of the year of this publication's release in 2022).
1 INTRODUCTION

1.1 OVERVIEW
1.2 WHAT IS LATENT DEMAND AND THE P.I.E.?
1.3 THE METHODOLOGY
  1.3.1 STEP 1. PRODUCT DEFINITION AND DATA COLLECTION
  1.3.2 STEP 2. FILTERING AND SMOOTHING
  1.3.3 STEP 3. FILLING IN MISSING VALUES
  1.3.4 STEP 4. VARYING PARAMETER, NON-LINEAR ESTIMATION
  1.3.5 STEP 5. FIXED-PARAMETER LINEAR ESTIMATION
  1.3.6 STEP 6. AGGREGATION AND BENCHMARKING
1.4 FREQUENTLY ASKED QUESTIONS (FAQ)
  1.4.1 CATEGORY DEFINITION
  1.4.2 UNITS
  1.4.3 METHODOLOGY

2 SUMMARY OF FINDINGS

2.1 LATENT DEMAND IN THE UNITED STATES
2.2 LATENT DEMAND BY YEAR IN THE UNITED STATES
2.3 TOP 100 CITIES IN THE UNITED STATES

3 FAR WEST

3.1 EXECUTIVE SUMMARY
3.2 LATENT DEMAND BY YEAR - ALASKA
3.3 CITIES SORTED BY RANK - ALASKA
3.4 LATENT DEMAND BY YEAR - CALIFORNIA
3.5 CITIES SORTED BY RANK - CALIFORNIA
3.6 LATENT DEMAND BY YEAR - HAWAII
3.7 CITIES SORTED BY RANK - HAWAII
3.8 LATENT DEMAND BY YEAR - NEVADA
3.9 CITIES SORTED BY RANK - NEVADA
3.10 LATENT DEMAND BY YEAR - OREGON
3.11 CITIES SORTED BY RANK - OREGON
3.12 LATENT DEMAND BY YEAR - WASHINGTON
3.13 CITIES SORTED BY RANK - WASHINGTON

4 GREAT LAKES

4.1 EXECUTIVE SUMMARY
4.2 LATENT DEMAND BY YEAR - ILLINOIS
4.3 CITIES SORTED BY RANK - ILLINOIS
4.4 LATENT DEMAND BY YEAR - INDIANA
4.5 CITIES SORTED BY RANK - INDIANA
4.6 LATENT DEMAND BY YEAR - MICHIGAN
4.7 CITIES SORTED BY RANK - MICHIGAN
4.8 LATENT DEMAND BY YEAR - OHIO
4.9 CITIES SORTED BY RANK - OHIO
4.10 LATENT DEMAND BY YEAR - WISCONSIN
4.11 CITIES SORTED BY RANK - WISCONSIN

5 MID-ATLANTIC

5.1 EXECUTIVE SUMMARY
5.2 LATENT DEMAND BY YEAR - DELAWARE
5.3 CITIES SORTED BY RANK - DELAWARE
5.4 LATENT DEMAND BY YEAR - DISTRICT OF COLUMBIA
5.5 CITIES SORTED BY RANK - DISTRICT OF COLUMBIA
5.6 LATENT DEMAND BY YEAR - MARYLAND
5.7 CITIES SORTED BY RANK - MARYLAND
5.8 LATENT DEMAND BY YEAR - NEW JERSEY
5.9 CITIES SORTED BY RANK - NEW JERSEY
5.10 LATENT DEMAND BY YEAR - NEW YORK
5.11 CITIES SORTED BY RANK - NEW YORK
5.12 LATENT DEMAND BY YEAR - PENNSYLVANIA
5.13 CITIES SORTED BY RANK - PENNSYLVANIA

6 NEW ENGLAND

6.1 EXECUTIVE SUMMARY
6.2 LATENT DEMAND BY YEAR - CONNECTICUT
6.3 CITIES SORTED BY RANK - CONNECTICUT
6.4 LATENT DEMAND BY YEAR - MAINE
6.5 CITIES SORTED BY RANK - MAINE
6.6 LATENT DEMAND BY YEAR - MASSACHUSETTS
6.7 CITIES SORTED BY RANK - MASSACHUSETTS
6.8 LATENT DEMAND BY YEAR - NEW HAMPSHIRE
6.9 CITIES SORTED BY RANK - NEW HAMPSHIRE
6.10 LATENT DEMAND BY YEAR - RHODE ISLAND
6.11 CITIES SORTED BY RANK - RHODE ISLAND
6.12 LATENT DEMAND BY YEAR - VERMONT
6.13 CITIES SORTED BY RANK - VERMONT

7 PLAINS

7.1 EXECUTIVE SUMMARY
7.2 LATENT DEMAND BY YEAR - IOWA
7.3 CITIES SORTED BY RANK - IOWA
7.4 LATENT DEMAND BY YEAR - KANSAS
7.5 CITIES SORTED BY RANK - KANSAS
7.6 LATENT DEMAND BY YEAR - MINNESOTA
7.7 CITIES SORTED BY RANK - MINNESOTA
7.8 LATENT DEMAND BY YEAR - MISSOURI
7.9 CITIES SORTED BY RANK - MISSOURI
7.10 LATENT DEMAND BY YEAR - NEBRASKA
7.11 CITIES SORTED BY RANK - NEBRASKA
7.12 LATENT DEMAND BY YEAR - NORTH DAKOTA
7.13 CITIES SORTED BY RANK - NORTH DAKOTA
7.14 LATENT DEMAND BY YEAR - SOUTH DAKOTA
7.15 CITIES SORTED BY RANK - SOUTH DAKOTA

8 ROCKIES

8.1 EXECUTIVE SUMMARY
8.2 LATENT DEMAND BY YEAR - COLORADO
8.3 CITIES SORTED BY RANK - COLORADO
8.4 LATENT DEMAND BY YEAR - IDAHO
8.5 CITIES SORTED BY RANK - IDAHO
8.6 LATENT DEMAND BY YEAR - MONTANA
8.7 CITIES SORTED BY RANK - MONTANA
8.8 LATENT DEMAND BY YEAR - UTAH
8.9 CITIES SORTED BY RANK - UTAH
8.10 LATENT DEMAND BY YEAR - WYOMING
8.11 CITIES SORTED BY RANK - WYOMING

9 SOUTHEAST

9.1 EXECUTIVE SUMMARY
9.2 LATENT DEMAND BY YEAR - ALABAMA
9.3 CITIES SORTED BY RANK - ALABAMA
9.4 LATENT DEMAND BY YEAR - ARKANSAS
9.5 CITIES SORTED BY RANK - ARKANSAS
9.6 LATENT DEMAND BY YEAR - FLORIDA
9.7 CITIES SORTED BY RANK - FLORIDA
9.8 LATENT DEMAND BY YEAR - GEORGIA
9.9 CITIES SORTED BY RANK - GEORGIA
9.10 LATENT DEMAND BY YEAR - KENTUCKY
9.11 CITIES SORTED BY RANK - KENTUCKY
9.12 LATENT DEMAND BY YEAR - LOUISIANA
9.13 CITIES SORTED BY RANK - LOUISIANA
9.14 LATENT DEMAND BY YEAR - MISSISSIPPI
9.15 CITIES SORTED BY RANK - MISSISSIPPI
9.16 LATENT DEMAND BY YEAR - NORTH CAROLINA
9.17 CITIES SORTED BY RANK - NORTH CAROLINA
9.18 LATENT DEMAND BY YEAR - SOUTH CAROLINA
9.19 CITIES SORTED BY RANK - SOUTH CAROLINA
9.20 LATENT DEMAND BY YEAR - TENNESSEE
9.21 CITIES SORTED BY RANK - TENNESSEE
9.22 LATENT DEMAND BY YEAR - VIRGINIA
9.23 CITIES SORTED BY RANK - VIRGINIA
9.24 LATENT DEMAND BY YEAR - WEST VIRGINIA
9.25 CITIES SORTED BY RANK - WEST VIRGINIA

10 SOUTHWEST

10.1 EXECUTIVE SUMMARY
10.2 LATENT DEMAND BY YEAR - ARIZONA
10.3 CITIES SORTED BY RANK - ARIZONA
10.4 LATENT DEMAND BY YEAR - NEW MEXICO
10.5 CITIES SORTED BY RANK - NEW MEXICO
10.6 LATENT DEMAND BY YEAR - OKLAHOMA
10.7 CITIES SORTED BY RANK - OKLAHOMA
10.8 LATENT DEMAND BY YEAR - TEXAS
10.9 CITIES SORTED BY RANK - TEXAS

11 DISCLAIMERS, WARRANTIES, AND USER AGREEMENT PROVISIONS

11.1 DISCLAIMERS & SAFE HARBOR
11.2 ICON GROUP INTERNATIONAL, INC. USER AGREEMENT PROVISIONS


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